The Minnesota Cystic Fibrosis Center recently gained membership to a research network for the disease, creating greater opportunities to treat patients in the Midwest and across the nation.
The center’s association with the Therapeutics Development Network will afford them greater access to new treatments and allow them to share test result information with other major institutions.
“This is the result of a rather profound and prolonged group effort. (The cystic fibrosis center) is one of the longest lived and most storied cystic fibrosis centers in the world,” Dr. David Cornfield, a fibrosis center physician, said.
Dr. Carlos Milla, director of the center, said being a part of the research network will have two major benefits for the center and its patients. It will increase the center’s involvement in developing new therapies and provide greater access to new treatments for the disease, including gene therapy.
The membership also makes it possible to test new drugs on a wider pool of patients.
Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said because only a small number of people have cystic fibrosis, institutions have to pool their resources to develop treatments.
Patient care at the center
ith approximately 450 patients – many of them from outside the state – the center is one of the nation’s largest.
Milla said the center’s patient population is particularly significant because the disease affects only one in 3,000 babies born in the state.
“It’s important to recognize that this is the only cystic fibrosis center in the upper Midwest, and as a result this will have an impact that reaches far beyond the walls of this facility,” Cornfield said.
The center was established in 1961 by University pediatrician Dr. Warren Warwick, who still works at the center.
Warwick has helped develop new treatments for the disease, such as the high-frequency chest compression vest.
In its first seven years, the mortality rate at the center dropped from 25 percent to 2 percent, where it remains. In addition, the median survival age for patients has increased from 2.5 years to more than 40 years.
Cornfield said the center uses an aggressive care plan to delay the onset of symptoms and slow the disease. He credits this approach for the center’s median survival rate, one of the longest in the world.
Cystic fibrosis is a genetic disease that affects the body’s mucus producing glands.
Healthy lungs should produce a large amount of mucus on a daily basis. But cystic fibrosis sufferers produce much less.
As a result, lung disease is the most serious ailment of those with cystic fibrosis. Mucus becomes trapped in the lungs and increases the risk for and seriousness of infections.
“Usually what happens is that they catch infections easily and those infections become chronic. So what for any of us would be a common cold, for these kids can end up being something more close to pneumonia,” Milla said.
The effect of the disease on the lungs can also lead to malnutrition and poor growth, Cornfield said.
Treatment for patients with cystic fibrosis has mostly focused on helping them manage symptoms of the disease with antibiotics and mucus-cleaning medications.
Milla said new therapies will go beyond treating the complications and examine the genetic defect that causes it.
Beall said the Therapeutic Development Network was established by the National Cystic Fibrosis Foundation in 1997 to discover alternative treatments such as gene therapy. The Minnesota Cystic Fibrosis Center is one of six institutions added to the research network this year, making a total of 14 in the network.
Beall said the research network needs to double the number of trials it conducts and triple its patient base in the coming years to keep up with the demand for new drugs.
Milla said he was excited by the recent developments in treating cystic fibrosis.
“Now we’re going to be able to treat the real problem and not just treat the consequences,” he said.
